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Could a pill replace gene therapy for sickle cell disease? Research uncovers potential path to small-molecule drugThey found that in order for BCL11A to be stable in cells and capable of shutting down fetal hemoglobin production, it must assemble into a tetramer—four connected BCL11A molecules. It does so ...
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MaxCyte: Building the Future of Cell and Gene Therapy InnovationMaxCyte's technology ensures that a large number of T cells are successfully edited with CRISPR/Cas-9 molecular scissors, which is critical for the effectiveness of the therapy. Casgevy enables CRISPR ...
in red blood cells, by introducing a gene known as BCL11A which down-regulates the production of the adult form of the oxygen-carrying molecule. Reverting to HbF in thalassaemia and SCD patients ...
The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...
that have been base-edited in the promotor regions of the HBG1/2 genes and are administered via a hematopoietic stem cell transplant procedure. The BEAM-101 edit is designed to inhibit the ...
Casgevy enables CRISPR/Cas-9 to edit the BCL11A gene in stem cells to produce fetal hemoglobin. The edited genes are infused into a patient's bone marrow to start producing red blood cells with ...
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